UniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, has announced that the company received final meeting minutes from the US Food and Drug Administration (FDA) regarding a pre-Biologics License Application (BLA) meeting held on 29 October last year to discuss the application for AMT-130, an investigational gene therapy for Huntington’s disease (HD).
In the final meeting minutes, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission. UniQure is carefully evaluating the feedback and plans to urgently request a follow-up meeting with the FDA to take place in the first quarter of 2026.
